Therapeutic Highlights from the 2022 WORLD Symposium
MPS I Orchard Therapeutics Announces Interim Data for OTL-203 Showing Positive Clinical Results in Multiple Disease Manifestations of Mucopolysaccharidosis Type I Hurler Syndrome (MPS-IH). Read the full report >> SiglionTherapeutics, Development of a Novel...
Connect With Rare Disease Communities!
Ultragenyx has relaunched their patient support interactive website, UltraRareAdvocacy.com with a refreshed design and more ways to connect with rare disease communities. Visitors will find: More rare experiences from individuals and families living with...
NICE Recommends Vimizim For Treating MPS IVA Morquio
We are excited to hear that Elosulfase alfa - branded as Vimizin and made by BioMarin - has been recommended by NICE (National Institute for Health and Care Excellence) for treating MPS IVA Morquio in England and Wales. Newly diagnosed patients and those already...
Recruiting for Clinical Trial: MPS-II (Hunter Syndrome) with Pabinafusp-Alfa
Mathias Schmidt, PhD, President and CEO of JCR Pharmaceuticals USA, discusses long-term efficacy and safety data of pabinafusp-alfa (Izcargo) in mucopolysaccharidosis type II (MPS II; Hunter syndrome). (Data from this study was recently presented...
Current Treatment Options Explained for Hurler Syndrome
(MPS I) Raymond Wang, MD, Metabolic Specialist and Director of the Multidisciplinary Lysosomal Storage Disorder Program at Children’s Hospital of Orange County, gives an overview of the treatment landscape for mucopolysaccharidosis type I. There are two MPS I...
Advocacy Groups Unveil ‘Roadmap’ for Sanfilippo Research, Care
A coalition of patient advocacy organizations has published a Global Roadmap for Sanfilippo Syndrome therapies, in an effort to accelerate research and develop effective therapies and care for families affected by Sanfilippo syndrome. “The Roadmap aims to capture the...
Rapid Test Expected to Help in Developing Treatments for Sanfilippo
A new study shows a 30-minute, inexpensive, and easy-to-use test may help measure the activity of NDST1, one of the main enzymes involved in heparan sulfate, the sugar molecule that build ups and causes Sanfilippo syndrome. Sanfilippo syndrome, or MPS III, is a...
Abeona Ends Enrollment For MPS IIIA Clinical Trial
We are disappointed to hear that Abeona Therapeutics has decided to end enrollment for their clinical trial (Study ABT-003) for MPS IIIA children aged 2-18 years with a Developmental Quotient score less than 60. The company has recently conducted a review of...
Resources for Courageous Parents
Courageous Parents Network is a non-profit organization and educational platform that orients, empowers and accompanies families and providers caring for children with serious illness. They have videos, podcasts, printable guides, Guided Pathways, and a blog to help...
The Rare Stars Award Ceremony
This will be held on the evening of Saturday, July 22, 2023 in conjunction with the National Family Conference. Head to our National Family Conference page for more details!